Clinical Trial with Cladribine (2-CDA) and Pegylated Interpheron Alfa-2a in patients with advanced systemic mastocytosis carrying D816V KIT mutation (or different mutations involving exon 17 of KIT).
- Conditions
- Advanced systemic mastocytosis carrying the D816V KIT mutation (or different exon 17 KIT mutations).Therapeutic area: Not possible to specify
- Registration Number
- EUCTR2012-000797-35-ES
- Lead Sponsor
- Hospital Virgen de la Salud, Toledo (Spain).
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- Not specified
Age older than 18 years.
Diagnosis of advanced systemic mastocytosis (aggressive systemic mastocytosis or proggressing systemic mastocytosis) with D816V or other exon 17 KIT mutations.
ECOG = 3.
Signed informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4
Impaired liver function (total bilirubin = 2.0 mg/dl, AST or ALT > 3 x upper limit of normal) not related to mastocytosis.
Impaired renal function (creatinine = 2.0 mg/dL) not related to mastocytosis.
Grade III-IV cytopenias not related to mastocytosis.
Severe cardiopathy (grade III/IV of NYHA, or left ventricular ejection fraction < 50%).
Pregnancy or breastfeeding.
Female patients who do not use contraceptive methods.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To analyze the efficacy and safety of the combined use of cladribine plus peginterpheron alfa-2a in patients with advanced systemic mastocytosis.;Secondary Objective: To analyze the safety of the combined use of cladribine plus peginterpheron alfa-2a in patients with advanced systemic mastocytosis.;Primary end point(s): To evaluate the effect of therapy on bone marrow mast cell infiltration.;Timepoint(s) of evaluation of this end point: 3 and 6 months.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.<br>To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis). <br>To determine the safety of combined therapy with cladribine plus pegylated interpheron alpha-2a. <br>To evaluate the effect of therapy on mastocytosis skin lesions. <br>To evaluate the effect of therapy on mastocytosis-related organomegalies.<br>To evaluate the effect of therapy on mastocytosis-related bone alterations.<br><br>;Timepoint(s) of evaluation of this end point: 6 months for every endpoint.